AML-MDS Novel Prognostic Tests Clinical Study

Completed

• Conditions: Myelodysplastic Syndrome (MDS); Acute Myeloid Leukemia (AML)

• Registration Number: NCT01685619
• Lead Sponsor: Nova Scotia Health Authority

Brief Summary

This clinical study will provide the study specimens (samples of bone marrow and blood) and the clinical data for a pan-Canadian collaborative research project developed by the MDS/AML Research Consortium. The goal of this project involves the evaluation and potential validation of five novel prognostic tests for myelodysplasia (MDS) and/or acute myeloid leukemia (AML), as well as an analysis of health economic and socio-ethical implications related to the potential introduction of these tests into the clinical setting. The over-arching goal is to improve the outcomes of patients with MDS and AML. The primary hypothesis is that one or more of the laboratory tests being evaluated in conjunction with this study, either alone or in combination with other laboratory tests (either established or under investigation in this project), will have statistically significant prognostic value either alone or in combination with established clinical risk factors.

The clinical study will involve the enrollment of 200 adults with AML and 200 adults with MDS over a 2.5 year period. Participants will be followed on study for two years. Bone marrow and blood specimens will be collected at diagnosis and at other time points as required for the development of the five laboratory tests.

Participants will be assigned to treatment according to local institutional practice and will be followed for up to 2 years. Health economic and quality of life questionnaires will be administered at key time points. Data will be collected regarding participant characteristics, diagnosis, disease features, treatment and clinical outcome.

Detailed Description

Two of the tests involve a technology called flow cytometry. Both of the flow cytometry tests are used to predict whether a person is likely to have a good response to chemotherapy or not.

Three of the tests involve new genetic-based technology. One of these tests is called comparative genomic profiling. This test can detect genetic abnormalities that current testing methods are not able to detect. Another test involves micro-RNA profiling. The final test involves RNA sequencing. The researchers think these tests might be useful in predicting how well a person will respond to treatment.

The novel laboratory tests being evaluated as part of this study are still in the early phases of development and cannot be used for clinical decision making. Participants enrolled in this study will not be informed regarding their individual results with respect to the study tests that are conducted using their biospecimens.

The following information (data) will be collected regarding study participants: diagnosis, results of relevant clinical tests, age, gender, treatment and outcome during the 2 year study follow-up period. The study also involves the completion of study questionnaires at six different time points over the course of the two year study follow-up.

Study Timeline

• Study First Submitted: 2012-09-12
• Study First Submitted QC: 2012-09-12
• Study First Posted: 2012-09-14
• Study Start: 2012-10-02
• Primary Completion: 2018-01-24
• Study Completion: 2018-01-24
• Status Verified: 2022-11
• Last Update Submitted: 2022-11-04
• Last Update Posted: 2022-11-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Prognostic capacity of the candidate tests (alone and in combination) to predict response to treatment, time to relapse, time to death.	Two years following the completion of enrollment.	For the AML cohort, the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, white blood cell count at presentation, antecedent hematologic disorder, FLT-3 status, Karnofsky Performance Status (KPS), cytogenetic sub-group (using WHO 2008). For the MDS cohort the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, KPS, karyotype, bone marrow blast count and number of cytopenias at diagnosis.

Secondary Outcome Measures

Name	Time	Method
Cost impact of candidate tests.	Two years following the completion of enrollment.	The cost-effectiveness of the candidate tests for AML and MDS treatment will be projected with the aid of economic simulation models.
Societal risks and benefits related to the candidate tests.	Two years following the completion of enrollment.	A comparative analysis of policies and regulations in Canada governing prognostic tests will be undertaken (including tests that utilize RNA and DNA based technologies). In addition, input will be obtained from key stakeholders. This information will be used to develop a set of guidelines and best practices for this type of research.

Trial Locations

Locations (6)

Vancouver General Hospital; 🇨🇦 Vancouver, British Columbia, Canada

Tom Baker Cancer Centre; 🇨🇦 Calgary, Alberta, Canada

CancerCare Manitoba; 🇨🇦 Winnipeg, Manitoba, Canada

Princess Margaret Cancer Centre (formerly Princess Margaret Hospital); 🇨🇦 Toronto, Ontario, Canada

Hôpital Maisonneuve-Rosemont; 🇨🇦 Montreal, Quebec, Canada

Queen Elizabeth II Health Sciences Centre; 🇨🇦 Halifax, Nova Scotia, Canada